Therapeutic/Technology: Small molecule therapy
Dual targeting of tyrosine kinase and BCL6 signaling for leukemia stem cell eradication
Leukemia is the most frequent form of cancer in children and teenagers, but is also common in adults. Chemotherapy has vastly improved the outcome of leukemia over the past four decades. However, many patients still die because of recurrence of the disease and development of drug-resistance in leukemia cells. In preliminary studies for this proposal […]
Dual targeting of tyrosine kinase and BCL6 signaling for leukemia stem cell eradication
Leukemia is the most frequent form of cancer in children and teenagers, but is also common in adults. Chemotherapy has vastly improved the outcome of leukemia over the past four decades. However, many patients still die because of recurrence of the disease and development of drug-resistance in leukemia cells. In preliminary studies for this proposal […]
New Drug Discovery for SMA using Patient-derived Induced Pluripotent Stem Cells
Spinal muscular atrophy (SMA) is the leading genetic cause of infant death in the U.S. This devastating disease affects 1 child in every 6,000-10,000 live births, with a North American prevalence of approximately 14,000 individuals. The disease is characterized by the death of spinal cord cells called motor neurons that connect the brain to muscle. […]
Cartilage Regeneration by the Chondrogenic Small Molecule PRO1 during Osteoarthritis
The ability to direct the differentiation of resident mesenchymal stem cells (MSCs) towards the cartilage lineage offers considerable promise for the regeneration of articular cartilage after traumatic joint injury or age-related osteoarthritis (OA). MSCs can be stimulated in vitro to form new functional cartilage. In the OA-affected joint, the repair is insufficient, leaving a damaged […]
Developing a drug-screening system for Autism Spectrum Disorders using human neurons
Autism and autism spectrum disorders (ASD) are complex neurodevelopmental diseases that affect 1 in 150 children in the United States. Such diseases are mainly characterized by deficits in verbal communication, impaired social interaction, and limited and repetitive interests and behavior. Because autism is a complex spectrum of disorders, a different combination of genetic mutations is […]
Preclinical development of a pan Bcl2 inhibitor for cancer stem cell directed therapy
Cancer is the leading cause of death for individuals under 85. Relapse and metastatic disease are the leading causes of cancer related mortality. Anti-apoptotic BCL2 family member overexpression has been shown to promote disease progression in both chronic myeloid leukemia (CML) and prostate cancer. Andr., the emergence of cancer stem cells (CSC) promotes apoptosis resistance […]
Stem Cell-mediated Therapy for High-grade Glioma: Toward Phase I-II Clinical Trials
Despite aggressive multimodal therapy and advances in imaging, surgical and radiation techniques, malignant brain tumors (high-grade gliomas) remain incurable, with survival often measured in months. Treatment failure is largely attributable to the diffuse and invasive nature of these brain tumor cells, ineffective delivery of chemotherapeutic agents to tumor sites, and toxic side-effects to the body, […]
Therapeutic Opportunities To Target Tumor Initiating Cells in Solid Tumors
Cancer is a major cause of human death worldwide. The vast majority of cancer patients suffer from solid tumors whose growth destroys vital organs. We propose to develop novel therapeutic drugs that target solid tumors affecting the brain, colon and ovaries. These cancers account for a significant proportion of currently intractable solid malignancies. Scientists have […]